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    Summary
    EudraCT Number:2021-002308-11
    Sponsor's Protocol Code Number:IM047-001
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-12-20
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2021-002308-11
    A.3Full title of the trial
    A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase 2/3, multicenter, randomized, double-blind study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of oral ozanimod (RPC1063) in pediatric subjects with moderately to severely active ulcerative colitis with an inadequate response to conventional therapy.
    A.4.1Sponsor's protocol code numberIM047-001
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1268-2191
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/233/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCelgene International II Sàrl
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCelgene Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBristol-Myers-Squibb International Corporation
    B.5.2Functional name of contact pointGSM-CT
    B.5.3 Address:
    B.5.3.1Street AddressParc de L'Alliance - Avenue de Finlande 4
    B.5.3.2Town/ cityBraine-l'Alleud
    B.5.3.3Post code1420
    B.5.3.4CountryBelgium
    B.5.6E-mailclinical.trials@bms.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOzanimod HCL
    D.3.2Product code RPC1063
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.23
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOzanimod HCL
    D.3.2Product code RPC1063
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.46
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 3
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOzanimod HCL
    D.3.2Product code RPC1063
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNOzanimod HCL
    D.3.9.1CAS number 1618636-37-5
    D.3.9.3Other descriptive nameRPC1063
    D.3.9.4EV Substance CodeSUB175614
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.92
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Moderately to Severely Active Ulcerative Colitis
    E.1.1.1Medical condition in easily understood language
    Ulcerative Colitis
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10045365
    E.1.2Term Ulcerative colitis
    E.1.2System Organ Class 100000004856
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical remission, Week 52)
    E.2.2Secondary objectives of the trial
    Evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical remission, Week 10)
    Evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical response, Week 52)
    Evaluate the efficacy of ozanimod in pediatric subjects with moderately to severely active UC (3-component Mayo Score: clinical response, Week 10)
    Evaluate symptomatic remission
    Evaluate time to achieve symptomatic remission
    Evaluate endoscopic improvement by Mayo Endoscopy Score
    Evaluate corticosteroid-free remission
    Evaluate the safety and tolerability of 2 doses of ozanimod in pediatric subjects w/moderately-severely active UC
    Characterize the pharmacokinetics of 2 doses of ozanimod and its major active metabolites in pediatric subjects with moderately-severely active UC
    Evaluate the pharmacodynamic effects of 2doses of ozanimod in pediatric subjects w/moderately-severely active UC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Age ≥ 2 and < 17 years of age, inclusive, (for the assigned cohort) at the time of informed
    consent/assent.
    Note: Subjects with a weight below the 5
    th percentile for age, should be discussed with
    the Medical Monitor prior to enrollment.
    2. One or both parent(s) or legal guardian(s) understands and voluntarily signs informed
    consent prior to any study-related assessments or procedures (as required by national or
    local regulations).
    - Assent from subjects will also be obtained in an age-appropriate manner in
    conjunction with applicable local regulations. Written assent templates, appropriate
    for relevant age ranges, will be made available to clinical study sites.
    3. Willing and able to adhere to the study visit schedule and other protocol requirements
    including swallowing a capsule (until a sprinkle formulation of ozanimod is available as
    described in Section 7.1).
    4. Have had UC diagnosed prior to the Screening Visit. The diagnosis should be confirmed
    by clinical and endoscopic evidence and corroborated by a histopathology report (note:
    histopathology may be performed during endoscopy at Screening if no prior report is
    readily available).
    5. Evidence of UC extending beyond the rectum, as determined by baseline endoscopy
    (flexible sigmoidoscopy or colonoscopy)
    6. Has moderately to severely active UC, defined as a 4-component Mayo Score ≥ 6 and
    ≤ 12 at the time of screening and day 1, including the following: Mayo Endoscopy Subscore ≥ 2,
    Rectal Bleeding Score ≥ 1, and Stool Frequency Score ≥ 1.
    7. Has had an inadequate response, loss of response to, or is intolerant to at least 1 of the
    following treatments for UC (Appendix B):
    a. oral aminosalicylates (eg, mesalamine, sulfasalazine, olsalazine, balsalazide) (N/A in France)
    b. systemic corticosteroids (eg, oral prednisone, budesonide MMX, IV corticosteroids)
    c. immunomodulators (eg, AZA, 6-MP, cyclosporine, MTX)
    d. biologic therapy (eg, abatacept, infliximab, etanercept, adalimumab, anakinra,
    rituximab, vedolizumab, and golimumab)
    e. other systemic immunomodulatory treatments for UC, such as tofacitinib
    Note: criterion 7b-7e are N/A in Germany
    8. Subjects may concurrently receive treatment with oral 5-ASAs, or with prednisone or
    equivalent (≤ 0.5 mg/kg/ day up to 20 mg/day) or beclomethasone < 5mg/day.
    - If on oral 5-ASAs, the dose must have been stable for at least 3 weeks prior to
    screening endoscopy = baseline .
    - If on corticosteroids, the dose must have been stable for at least 2 weeks prior to
    screening endoscopy and must remain stable through the first 5 weeks of treatment
    (ie, until the Week 5 study visit).
    -If discontinuing corticosteroids (e.g. MMX), subject must taper as described in Section 8.1.1.
    9. Inclusion criterion 9 is not applicable for protocol amendment 2.0.
    10. Must have documentation of vaccinations per standard immunization schedule including
    complete Varicella vaccination at least 30 days prior to randomization. (VZV and IgG +ve)
    11. Females of childbearing potential (FCBP)1 must agree to practice a highly effective
    method of contraception2
    throughout the study until completion of the 90-day Safety
    Follow-up Visit. Highly effective methods of contraception are those that alone or in
    combination result in a failure rate of a Pearl index of less than 1% per year when used
    consistently and correctly. Selection of contraceptive methods should be based on those
    available/approved as per national or local practice. Acceptable methods of birth control
    in the study are the following:
    - combined hormonal (estrogen and progestogen containing) contraception, which may
    be oral, intravaginal, or transdermal
    - progestogen-only hormonal contraception associated with inhibition of ovulation,
    which may be oral, injectable, or implantable
    - placement of an intrauterine device (IUD)
    - placement of an intrauterine hormone-releasing system (IUS)
    - bilateral tubal occlusion
    - complete sexual abstinence ([defined as refraining from heterosexual intercourse]
    reliability to be evaluated based on the preferred and usual lifestyle of the subject)
    - Periodic abstinence (calendar, symptothermal, post-ovulation methods),
    withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method
    E.4Principal exclusion criteria
    Cardiovascular, hepatic, neurological, pulmonary, ophthalmological, endocrine, psychiatric, or other major systemic disease making implementation of the protocol or interpretation of the study difficult or that would put the subject at risk by participating in the study
    Crohn’s disease, indeterminate colitis or the presence or history of a fistula consistent with Crohn’s disease or microscopic colitis, radiation colitis or ischemic colitis
    +ve for toxin producing Clostridium difficile or PCR exam of stool. If +ve, subjects may be rescreened after appropriate treatment and retested no earlier than 7 days after treatment completion
    +ve examination for pathogens. If positive, subjects may be treated and rescreened
    History of treatment with topical rectal 5-aminosalicylic acid or topical rectal steroids within 2 weeks of Screening endoscopy or anti-motility medications
    Apheresis within 2 weeks of randomization
    Delayed growth or pubertal development and who will not maintain a stable dose of corticosteroids through Week 5
    Pregnancy (also on day 1), lactation, or positive serum β-hCG during Screening
    History or presence of: Structural cardiac disease, Cardiac events or diseases that predispose to cardiac complications, Prolonged QT interval corrected for heart rate using Fridericia’s formula>450 msec for both genders, or additional risk for QT interval prolongation, resting HR < 55 bpm in subjects 12-17 years old, resting HR < 65 bpm in subjects 6-11 years old, or resting HR < 75 bpm in subjects 2-5 years old. Subjects allowed one recheck per Visit, untreated sleep apnea
    History of DM type 1, or uncontrolled DM type 2 glycosylated hemoglobin (HbA1c > 9%) or is a diabetic subject with significant comorbid conditions
    History of uveitis within the last year prior or at Screening, or history or evidence of retinal disease
    Subject has any known active bacterial, viral, fungal, mycobacterial infection, or any major episode infection that required hospitalization or treatment with intravenous antibiotics within 30 days of Screening or oral antibiotics within 14 days of screening
    In the case of prior SARS-CoV-2 infection, symptoms must be resolved and based on Investigator assessment, there are no sequelae that would place the subject at a higher risk of receiving investigational treatment
    Recurrent or chronic infection, recurrent urinary tract infections are allowed, there should also be documented evidence of surveillance for dysplasia for all subjects with left-sided colitis of > 12 years’ duration and total or extensive colitis of > 8 years’ duration.
    History of cancer
    History of or currently active primary or secondary immunodeficiency, or subjects with known genetic disorders as a cause for colitis
    ECG showing clinically significant abnormality
    Serum creatinine > 1.4 mg/dL for females, or > 1.6 mg/dL for males
    Liver function impairment; or, persisting elevations of alanine aminotransferase or aspartate aminotransferase > 2x upper limit of normal; or direct bilirubin > 1.5x UL
    Platelet < 100,000/μL
    Hemoglobin 7 < 8 g/dL
    Neutrophil < 1500/μL
    Absolute white blood < 3500/μL
    ALC < 800/μL
    Stool positive for pathogens
    Pulmonary function test measurements: Forced expiratory volume at 1 second or a forced vital capacity < 70% of predicted values, Hypersensitivity to active ingredients or excipients of ozanimod
    Treated with biological or investigational agent, including for SARS-CoV-2, within 4 weeks of that agent prior to the first dose of IP. Undetectable drug levels on therapeutic drug monitoring assays may be utilized
    Received live attenuated vaccine within 4 weeks prior to the first dose of IP. Non-live vaccinations can be administered during the study
    Any previous treatment with lymphocyte-depleting therapies
    Treatment with D-penicillamine, leflunomide, or thalidomide within 8 weeks of first dose of IP
    Previous treatment with natalizumab, fingolimod, or other S1P receptor modulators
    History of treatment with IV immune globulin, or plasmapheresis within 3 months prior to first dose of IP
    Previous treatment with oral cyclosporine, tacrolimus, sirolimus, or mycophenolate mofetil within 16 weeks of initiation of Screening
    Planned concurrent treatment with immunomodulatory after randomization. Subjects receiving AZA, 6-MP, or MTX at screening must discontinue treatment with these agents prior to first dose of IP
    Chronic nonsteroidal anti-inflammatory drug use (occasional use of NSAIDs, acetaminophen and aspirin PRN or up to 325 mg/day is permitted)
    Receiving treatment with Class Ia or Class III anti-arrhythmic drugs or with 2 or more agents in combination known to prolong PR interval
    Received cholestyramine or other drugs interfering with enterohepatic circulation within 3 weeks of Screening
    Receiving treatment with:
    At randomization:
    Inducers
    2 weeks prior to randomization:
    Monoamine oxidase inhibitors
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects who achieve clinical remission, defined as all the following:
    • Mayo Endoscopy Subscore (MES) ≤ 1
    • Rectal Bleeding Subscore (RBS) = 0
    • Stool Frequency Subscore (SFS) ≤ 1, with a decrease of ≥ 1 point from Baseline SFS

    Proportion of subjects who achieve histological remission, defined as the following:
    - Geboes index score < 2.0
    - Robarts Histopathology Index (RHI) ≤ 3, with subscores of 0 for both lamina propria
    neutrophils and neutrophils in the epithelium

    Early Discontinuation: Starting at Week 5 or later if either one of the following criteria are met
    at 2 consecutive visits that are at least 2 weeks apart:
    - Partial Mayo score is ≥ 7 and is the same or worse than baseline, AND rectal bleeding and
    stool frequency are not improved from baseline
    - Partial Mayo score is 6 and worse than baseline, AND rectal bleeding and stool frequency
    are not improved from baseline
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    E.5.2Secondary end point(s)
    1/Proportion of subjects who achieve clinical response defined as all the following:
    • Decrease from Baseline in the 3-component Mayo score of at least 2 points and at least 35%
    • Decrease in RBS of at least 1 point OR absolute RBS ≤ 1
    2/Proportion of subjects who achieve clinical remission defined as all the following:
    • MES ≤ 1
    • RBS = 0
    • SFS ≤ 1, with a decrease of ≥1 point from Baseline SFS
    3/Proportion of subjects who achieve symptomatic improvement of UC, defined as all the following:
    • RBS = 0
    • SFS ≤ 1
    • Decrease in SFS of ≥ 1 from Baseline
    4/Proportion of subjects who achieve endoscopic improvement, defined as MES ≤ 1
    5/Proportion of subjects who achieve corticosteroid-free remission at Week 52, defined as all the following:
    • Did not receive steroids for ≥ 12 weeks prior to Week 52
    • Achieved clinical remission defined by the 3-component Mayo score
    6/Secondary – Safety and Tolerability
    Number and proportion of subjects experiencing AEs, SAEs, AEs leading to discontinuation from treatment, and AEs of interest (AEIs)
    7/Secondary - Pharmacokinetics
    Steady state systemic exposure of ozanimod and CC112273
    8/Secondary – Pharmacodynamics
    Absolute and percent change from baseline in ALC
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/Week 10 and week 52
    2/Week 10
    3/Week 10 and week 52
    4/Week 10 and week 52
    5/Week 52
    6/throughout the study
    7/Week 18 and throughout the study
    8/Week 10 and week 52
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Israel
    Japan
    Russian Federation
    United Kingdom
    United States
    Belgium
    France
    Germany
    Netherlands
    Poland
    Spain
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    Last patient Last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days25
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months7
    E.8.9.2In all countries concerned by the trial days7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 120
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 48
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 72
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 120
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Subjects who continue to demonstrate clinical benefit will be eligible to receive Celgene supplied study treatment for a maximum treatment duration as specified in Section 7.1. of the protocol.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2023-02-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-05-04
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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